EP3589290A4 - Translatable molecules and synthesis thereof - Google Patents

Translatable molecules and synthesis thereof Download PDF

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Publication number
EP3589290A4
EP3589290A4 EP18761078.7A EP18761078A EP3589290A4 EP 3589290 A4 EP3589290 A4 EP 3589290A4 EP 18761078 A EP18761078 A EP 18761078A EP 3589290 A4 EP3589290 A4 EP 3589290A4
Authority
EP
European Patent Office
Prior art keywords
synthesis
translatable molecules
translatable
molecules
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP18761078.7A
Other languages
German (de)
English (en)
French (fr)
Other versions
EP3589290A1 (en
Inventor
Pattraranee LIMPHONG
Kiyoshi Tachikawa
Padmanabh Chivukula
Daiki MATSUDA
Arisa CALE
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Arcturus Therapeutics Inc
Original Assignee
Arcturus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Arcturus Therapeutics Inc filed Critical Arcturus Therapeutics Inc
Publication of EP3589290A1 publication Critical patent/EP3589290A1/en
Publication of EP3589290A4 publication Critical patent/EP3589290A4/en
Pending legal-status Critical Current

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Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • C07K14/505Erythropoietin [EPO]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/1816Erythropoietin [EPO]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482Serine endopeptidases (3.4.21)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482Serine endopeptidases (3.4.21)
    • A61K38/4846Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575Hormones
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81Protease inhibitors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
EP18761078.7A 2017-02-28 2018-02-27 Translatable molecules and synthesis thereof Pending EP3589290A4 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762465073P 2017-02-28 2017-02-28
PCT/US2018/020018 WO2018160592A1 (en) 2017-02-28 2018-02-27 Translatable molecules and synthesis thereof

Publications (2)

Publication Number Publication Date
EP3589290A1 EP3589290A1 (en) 2020-01-08
EP3589290A4 true EP3589290A4 (en) 2020-12-30

Family

ID=63370513

Family Applications (1)

Application Number Title Priority Date Filing Date
EP18761078.7A Pending EP3589290A4 (en) 2017-02-28 2018-02-27 Translatable molecules and synthesis thereof

Country Status (4)

Country Link
US (2) US11407800B2 (https=)
EP (1) EP3589290A4 (https=)
JP (3) JP7674077B2 (https=)
WO (1) WO2018160592A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11407800B2 (en) 2017-02-28 2022-08-09 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof
AU2019394996B2 (en) 2018-12-06 2025-11-06 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
CN110331147A (zh) * 2019-06-21 2019-10-15 苏州吉玛基因股份有限公司 一种mRNA的制备方法及其在肿瘤治疗中的应用
US12351834B2 (en) 2020-03-03 2025-07-08 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
AU2021236068A1 (en) 2020-03-09 2022-10-06 Arcturus Therapeutics, Inc. Compositions and methods for inducing immune responses
CA3176844A1 (en) 2020-05-01 2021-11-04 Carlos G. PEREZ-GARCIA Nucleic acids and methods of treatment for cystic fibrosis
EP4196128A4 (en) 2020-08-14 2024-06-12 Arcturus Therapeutics, Inc. METHOD FOR FREEZE DILIZATION OF LIPID NANOPARTICLES
CA3128660A1 (en) * 2020-10-09 2022-04-09 Eric G. Marcusson Compositions and methods for the prevention and/or treatment of covid-19
CN113372432A (zh) * 2021-06-15 2021-09-10 深圳市臻质医疗科技有限公司 一种基于化学修饰mRNA编码蛋白因子诱导和/或增强软骨损伤修复的方法
KR102785258B1 (ko) * 2022-09-07 2025-03-26 아이진 주식회사 변형핵산 함유 mRNA의 체내 전달용 조성물
EP4356933A1 (en) * 2022-09-07 2024-04-24 Eyegene, Inc. Composition for delivering modified nucleic acid-containing mrna

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016070166A2 (en) * 2014-11-02 2016-05-06 Arcturus Therapeutics, Inc. Messenger una molecules and uses thereof
US20160237134A1 (en) * 2014-11-10 2016-08-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof

Family Cites Families (19)

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ES2340499T3 (es) 2001-06-05 2010-06-04 Curevac Gmbh Arnm de antigeno tumoral estabilizado con un contenido de g/c aumentado.
AU2002338926B2 (en) * 2001-11-05 2007-05-24 Janssen Pharmaceutica N.V. Method for the in vitro synthesis of short double stranded RNAs
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
US8304529B2 (en) 2006-07-28 2012-11-06 Life Technologies Corporation Dinucleotide MRNA cap analogs
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
US20090226906A1 (en) * 2008-03-05 2009-09-10 Helicos Biosciences Corporation Methods and compositions for reducing nucleotide impurities
DE19216461T1 (de) 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
US9283287B2 (en) * 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
KR102121086B1 (ko) * 2012-11-01 2020-06-09 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
PL2922554T3 (pl) * 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
KR102354389B1 (ko) * 2013-08-21 2022-01-20 큐어백 아게 Rna―암호화된 단백질의 발현을 증가시키는 방법
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
JP6506749B2 (ja) 2013-10-22 2019-04-24 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのmRNA療法
GB201403096D0 (en) * 2014-02-21 2014-04-09 Oxford Nanopore Tech Ltd Sample preparation method
WO2016077123A1 (en) * 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Multiparametric nucleic acid optimization
WO2017201317A1 (en) * 2016-05-18 2017-11-23 Modernatx, Inc. Polyribonucleotides containing reduced uracil content and uses thereof
US11407800B2 (en) 2017-02-28 2022-08-09 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016070166A2 (en) * 2014-11-02 2016-05-06 Arcturus Therapeutics, Inc. Messenger una molecules and uses thereof
US20160237134A1 (en) * 2014-11-10 2016-08-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof

Also Published As

Publication number Publication date
WO2018160592A1 (en) 2018-09-07
JP2023052106A (ja) 2023-04-11
JP2026016392A (ja) 2026-02-03
JP7674077B2 (ja) 2025-05-09
US11407800B2 (en) 2022-08-09
US20180327471A1 (en) 2018-11-15
JP2020510426A (ja) 2020-04-09
US11939363B2 (en) 2024-03-26
EP3589290A1 (en) 2020-01-08
US20230059111A1 (en) 2023-02-23

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